Umbilical cord blood (CB) has been an alternative hematopoietic stem cell source especially for patients without an appropriate marrow or mobilized peripheral blood donor. Although many studies have shown similar overall survival rates after CB transplantation compared with other donor sources, higher rate of non-relapse mortality is a major obstacle for a successful CB transplantation. Thus, selecting a best appropriate unit is very important to improve the outcome of CB transplantation. Adequate cell dose and better HLA matching (antigen-level for -A, -B, and allele-level for -DRB1) have been considered most important criteria of donor choice for CB transplantation. In recent, other criteria including non-inherited maternal antigens, HLA-C matching, allele-level matching at 8 loci (-A, -B, -C, -DRB1), and anti-HLA antibodies are also suggested as factors that might affect the outcome of CB transplantation. This review will highlight current issues regarding criteria of donor choice for CB transplantation. Finally, I will introduce the algorithm and detailed guideline for CB selection recently developed in Korea, which may help physicians to choose best unit available.
Antibodies ; Blood Donors ; Bone Marrow ; Fetal Blood* ; Hematopoietic Stem Cell Transplantation* ; Hematopoietic Stem Cells* ; HLA-C Antigens ; Humans ; Korea ; Mortality ; Survival Rate ; Tissue Donors

Hematopoietic stem cell transplantation (HSCT) is the first field where human stem cell therapy was successful. Flooding interest on human stem cell therapy to cure previously incurable diseases is largely indebted to HSCT success. Allogeneic HSCT has been an important modality to cure various diseases including hematologic malignancies, various non-malignant hematologic diseases, primary immunodeficiency diseases, and inborn errors of metabolism, while autologous HSCT is generally performed to rescue bone marrow aplasia following high-dose chemotherapy for solid tumors or multiple myeloma. Recently, HSCs are also spotlighted in the field of regenerative medicine for the amelioration of symptoms caused by neurodegenerative diseases, heart diseases, and others. Although the demand for HSCs has been growing, their supply often fails to meet the demand of the patients needing transplant due to a lack of histocompatible donors or a limited cell number. This review focuses on the generation and large-scale expansion of HSCs, which might overcome current limitations in the application of HSCs for clinical use. Furthermore, current proof of concept to replenish hematological homeostasis from non-hematological origin will be covered.
Bone Marrow ; Cell Count ; Drug Therapy ; Heart Diseases ; Hematologic Diseases ; Hematologic Neoplasms ; Hematopoietic Stem Cell Transplantation ; Hematopoietic Stem Cells* ; Homeostasis ; Humans ; Metabolism, Inborn Errors ; Multiple Myeloma ; Neurodegenerative Diseases ; Regenerative Medicine ; Stem Cells ; Tissue Donors

BACKGROUND: Wilms tumor, the most common primary malignant renal tumor of childhood, has relatively good prognosis among solid tumors occurring in childhood because of the improved operation skill, proper selection of chemotherapeutic agents, and combined radiotherapy on the primary and distant metastatic sites after nephrectomy. However, successful therapy has been associated with long-term toxicity occurring years or decades later. So it is important to identify and understand the possible late effects of treatment for Wilms tumor. METHODS: We reviewed 73 cases of Wilms tumor who were diagnosed and treated from Mar., 1983 to Nov., 1996 and calculated the survival rate. In addition, 42 cases were investigated to see the late effects of treatment for Wilms tumor since Feb., 1993, especially with predesigned indices such as blood pressure, blood urea nitrogen(BUN), serum creatinine(S-Cr), 24 hour urine protein/creatinine(24 HU Pr/Cr), 24 hour urine microalbumin(24 HU MA), 24 hour urine beta2-microglobulin/creatinine(24 HU beta2-MG/Cr). RESULTS: Overall survival rate of Wilms tumor was 86% in 5 years. There was significant difference in survival between stage I, II, III group and stage IV, V group(90% vs 72%, P=0.032), and survival rate of favorable histology group was higher than that of unfavorable histology group(92% vs 73%, P=0.043). Of the 42 cases in our study of late effects, no patient has presented significant late sequelae causing morbidity, and there were one case of transient hypertension, three cases of microalbuminuria, three cases of proteinuria, one case of asymptomatic microscopic hematuria, one case of radiation hepatitis and two cases of nut-cracker syndrome. No abnormality was noted in BUN, S-Cr, or 24 HU beta2-MG/Cr. CONCLUSION: Wilms tumor in our center have an excellent prognosis and no significant late sequelae that might affect long-term morbidity were found. But thorough and further longer follow-up is mandatory to understand the possible late effects fully, so that cope with properly and improve the quality of life.
Blood Pressure ; Follow-Up Studies ; Hematuria ; Hepatitis ; Humans ; Hypertension ; Nephrectomy ; Prognosis ; Proteinuria ; Quality of Life ; Radiotherapy ; Survival Rate* ; Urea ; Wilms Tumor*

In the initial published version of this article, there was a mistake in the title. The correct title should be “Impact of Day 14 Peripheral Blood Chimerism after Allogeneic Hematopoietic Stem Cell Transplantation on the Treatment Outcome of Non-Malignant Disease”.

PURPOSE: This study was performed to compare the clinical characteristics of 2009 pandemic influenza A(H1N1) [A(H1N1) pdm09] and seasonal influenza A infection in the pediatric cancer patients. METHODS: A retrospective review was performed in the pediatric cancer patients who had confirmed A(H1N1)pdm09 infection at Samsung Medical Center from August 2009 to February 2010. For the comparison, the medical records of pediatric cancer patients with seasonal influenza A from January 2000 to May 2009 were reviewed retrospectively. RESULTS: Eighty-two A(H1N1)pdm09 infections were confirmed in the pediatric cancer patients. Ten patients (12.2%) developed complicated clinical course by lower respiratory infections or extrapulmonary infections; 4 pneumonia, 1 bronchitis, 1 pericarditis with pneumonia, 1 encephalitis with pneumonia, 2 meningitis and 1 pericarditis. Three patients received mechanical ventilator and ICU care. Three pediatric cancer patients (3.7%) died. The risk factors related to complicated A(H1N1)pdm09 infections were date of infection (44-45th week 2009) and nosocomial infection. When comparing with previous seasonal influenza A infections, more prompt and aggressive antiviral therapy was given in A(H1N1)pdm09 infections. CONCLUSION: The A(H1N1)pdm09 infections caused a various clinical manifestations including fatal cases in pediatric cancer patient during pandemic season. There was no significant difference in clinical course between influenza A(H1N1)pdm09 and seasonal influenza A infections except the antiviral treatment strategy.
Bronchitis ; Child ; Cross Infection ; Encephalitis ; Humans ; Influenza, Human ; Medical Records ; Meningitis ; Pandemics ; Pericarditis ; Pneumonia ; Respiratory Tract Infections ; Retrospective Studies ; Risk Factors ; Seasons ; Ventilators, Mechanical

Purpose: Children with Down syndrome (DS) show a higher risk of acute leukemia than those without DS. In this study, we investigated the nationwide incidence of acute leukemia among children with DS and compared their epidemiologic characteristics with those of children with acute leukemia but without DS. Materials and Methods: Using the National Health Insurance Service database, we selected patients with acute leukemia aged 0–19 years at diagnosis between 2007 and 2016. Results: Among the 4,697 children with acute leukemia, 54 (1.1%) had DS. The median incidence rate of leukemia with DS by year was 1.3% (range, 0.2%–2.0%). Sixteen patients with acute lymphoblastic leukemia (ALL; 29.6%) and 36 with acute myeloid leukemia (AML; 66.7%) had DS. The DS group showed younger age at diagnosis than the non-DS group, and diagnosis of AML was more frequent in the DS group than in the non-DS group (3 years vs. 9 years, p<0.001; 66.7% vs. 32.4%, P<0.001, respectively). The 5-year overall survival was comparable between the DS and non-DS groups (88.0% vs. 81.9%, p=0.375). Among all the Koreans born between 2007 and 2008, the incidences of acute leukemia, ALL, and AML were 49.25, 20.75, and 163.38 times higher, respectively, in the DS group than in the non-DS group. Conclusion Our findings support the fact that the incidence of acute leukemia is higher among patients with DS than among those without DS in Korea. However, the DS and non-DS groups in this study had a comparable overall survival rate.

Purpose: Recent treatments for pediatric acute lymphoblastic leukemia (ALL) are founded on risk stratification. We examined the survival rates and prognostic factors of patients over a 20-year period at a single institution. Materials and Methods: This study analyzed patients diagnosed with ALL and treated at the Pediatric Department of Samsung Medical Center (SMC). Patients were categorized into standard-risk (SR), high-risk (HR), and very high-risk (VHR) groups. The SMC protocol for the HR group underwent two changes during the study period: a modified Children’s Cancer Group (CCG)-1882 protocol was used from 2000 to 2005, the Korean multicenter HR ALL-0601 protocol from 2006 to 2014, and the Korean multicenter HR ALL-1501 protocol from 2015 to 2019. Results: Of the 460 patients, complete remission was achieved in 436 patients (94.8%). The 10-year overall survival rate (OS) was 83.8±1.9% for all patients. OS according to the SMC risk group was as follows: 95.9%±1.4% in the SR group, 83.8%±3.6% in the HR group, and 66.2%±6.9% in the VHR group. The 5-year OS within the HR group varied according to the treatment protocol: 73.9%±7.5%, in the modified CCG-1882 protocol, 83.0%±3.9%, in the 0601 protocol, and 96.2%±2.6%, in the 1501 protocol. For those aged 15 years and older, the OS was only 56.5%±13.1%. Relapse occurred in 71 patients (15.4%), and the OS after relapse was 37.7%±6.0%. Conclusion The treatment outcomes of patients with ALL improved markedly. However, there is a need to further characterize adolescents and young adult patients, as well as those who have experienced relapses.

Purpose: Recent treatments for pediatric acute lymphoblastic leukemia (ALL) are founded on risk stratification. We examined the survival rates and prognostic factors of patients over a 20-year period at a single institution. Materials and Methods: This study analyzed patients diagnosed with ALL and treated at the Pediatric Department of Samsung Medical Center (SMC). Patients were categorized into standard-risk (SR), high-risk (HR), and very high-risk (VHR) groups. The SMC protocol for the HR group underwent two changes during the study period: a modified Children’s Cancer Group (CCG)-1882 protocol was used from 2000 to 2005, the Korean multicenter HR ALL-0601 protocol from 2006 to 2014, and the Korean multicenter HR ALL-1501 protocol from 2015 to 2019. Results: Of the 460 patients, complete remission was achieved in 436 patients (94.8%). The 10-year overall survival rate (OS) was 83.8±1.9% for all patients. OS according to the SMC risk group was as follows: 95.9%±1.4% in the SR group, 83.8%±3.6% in the HR group, and 66.2%±6.9% in the VHR group. The 5-year OS within the HR group varied according to the treatment protocol: 73.9%±7.5%, in the modified CCG-1882 protocol, 83.0%±3.9%, in the 0601 protocol, and 96.2%±2.6%, in the 1501 protocol. For those aged 15 years and older, the OS was only 56.5%±13.1%. Relapse occurred in 71 patients (15.4%), and the OS after relapse was 37.7%±6.0%. Conclusion The treatment outcomes of patients with ALL improved markedly. However, there is a need to further characterize adolescents and young adult patients, as well as those who have experienced relapses.

Purpose: Recent treatments for pediatric acute lymphoblastic leukemia (ALL) are founded on risk stratification. We examined the survival rates and prognostic factors of patients over a 20-year period at a single institution. Materials and Methods: This study analyzed patients diagnosed with ALL and treated at the Pediatric Department of Samsung Medical Center (SMC). Patients were categorized into standard-risk (SR), high-risk (HR), and very high-risk (VHR) groups. The SMC protocol for the HR group underwent two changes during the study period: a modified Children’s Cancer Group (CCG)-1882 protocol was used from 2000 to 2005, the Korean multicenter HR ALL-0601 protocol from 2006 to 2014, and the Korean multicenter HR ALL-1501 protocol from 2015 to 2019. Results: Of the 460 patients, complete remission was achieved in 436 patients (94.8%). The 10-year overall survival rate (OS) was 83.8±1.9% for all patients. OS according to the SMC risk group was as follows: 95.9%±1.4% in the SR group, 83.8%±3.6% in the HR group, and 66.2%±6.9% in the VHR group. The 5-year OS within the HR group varied according to the treatment protocol: 73.9%±7.5%, in the modified CCG-1882 protocol, 83.0%±3.9%, in the 0601 protocol, and 96.2%±2.6%, in the 1501 protocol. For those aged 15 years and older, the OS was only 56.5%±13.1%. Relapse occurred in 71 patients (15.4%), and the OS after relapse was 37.7%±6.0%. Conclusion The treatment outcomes of patients with ALL improved markedly. However, there is a need to further characterize adolescents and young adult patients, as well as those who have experienced relapses.

PURPOSE: Recently Wilms tumor gene (WT1) transcripts have been detected in leukemia regardless of the disease subtype and the specific DNA markers suggesting that WT1 gene might be a useful panleukemic marker for monitoring minimal residual disease (MRD). This study was performed to investigate the expression of WT1 gene by a quantitative methods and to find the prognostic value of WT1 gene in childhood acute leukemia. METHODS: From the mononuclear cells isolated from bone marrow aspirates and peripheral bloods of 22 childhood acute and chronic leukemia patients, mRNA were extracted for the reverse transcriptase-polymerase chain reactions (RT-PCR). Relative levels of WT1 gene expression was calculated by using the value in K562 cell line to be 1.00 as a positive control. RESULTS: The sensitivity of detection of MRD with WT1 primers was 10 4 and comparable to that of bcr/abl expression in K562 cells and a patient with CML in blast crisis. WT1 gene expression was detected in 17 of 22 (77%) patients; 9/10 of acute lymphoblastic leukemia (ALL), 6/10 acute myelogenous leukemia (AML), 1 acute mixed lineage leukemia (AMLL) and 1 chronic myelogenous leukemia (CML) in blast crisis. In 4 AML patients who received autologous peripheral blood stem cell transplantation (PBSCT), two patients relapsed after reappearance of WT1 gene expression in bone marrow aspirates and the remaining two were in complete remission without expression of WT1 gene. CONCLUSION: These results show that WT1 gene expression is frequently noted in childhood acute leukemia and can be a useful sensitive marker for the detection of MRD comparable to bcr/abl transcripts. WT1 gene can be used as a panleukemic marker for the MRD monitoring for the evaluation of the remission status and in predicting early relapse in children with acute leukemia in the molecular levels. It may also be a useful tool for the detection of leukemic cell contamination in the process of peripheral blood stem cell transplantation.
Blast Crisis ; Bone Marrow ; Cell Line ; Child ; Gene Expression ; Genetic Markers ; Humans ; K562 Cells ; Leukemia* ; Leukemia, Myelogenous, Chronic, BCR-ABL Positive ; Leukemia, Myeloid, Acute ; Neoplasm, Residual ; Peripheral Blood Stem Cell Transplantation ; Precursor Cell Lymphoblastic Leukemia-Lymphoma ; Recurrence ; RNA, Messenger ; Wilms Tumor